title: How to treat patients with systemic amyloid light chain amyloidosis? Comparison of high-dose melphalan, low-dose chemotherapy and no chemotherapy in patients with or without cardiac amyloidosis.
abstract: BACKGROUND
  High-dose melphalan and autologous stem cell transplantation (HDM) is an effective treatment for systemic amyloid light chain (AL) amyloidosis but the eligibility criteria exclude many patients with this disorder. The aim of this study was to determine appropriate treatment strategies for systemic AL amyloidosis according to each patient's clinical condition in Japan.
  
  
  METHODS
  Historical cohort study. Fifty-three patients with systemic AL amyloidosis (those with malignancies were excluded) were treated in our hospital with HDM (15 patients), melphalan + prednisolone (MP) (17 patients), vincristine + adriamycin + dexamethasone (VAD) (11 patients), or supportive treatment (no chemotherapy, 10 patients). We compared the survival rates among these treatment groups.
  
  
  RESULTS
  Mean survival was significantly longer in the HDM group than in the other three groups (P < 0.01, log-rank test). This trend remained the same when patients were divided into those with and without cardiac amyloid involvement. Furthermore, in patients with heart involvement, survival in the VAD therapy group was significantly inferior to that in the MP therapy group (P < 0.01 by log-rank test). Significant factors related to the survival rate included the presence or absence of heart involvement and treatment modality.
  
  
  CONCLUSIONS
  HDM should be considered the treatment of choice in eligible patients with systemic AL amyloidosis even in the presence of cardiac amyloidosis. If HDM is not eligible, indications for VAD therapy should be carefully evaluated in patients with cardiac amyloidosis.
authors.affiliation: Nephrology Center, Toranomon Hospital, 2-2-2, Toranomon, Minato-ku, Tokyo 105-8470, Japan. hoshino@toranomon.gr.jp
authors.forename: Junichi
authors.initials: J
authors.lastname: Hoshino
authors.forename: Yoshifumi
authors.initials: Y
authors.lastname: Ubara
authors.forename: Naoki
authors.initials: N
authors.lastname: Sawa
authors.forename: Keiichi
authors.initials: K
authors.lastname: Sumida
authors.forename: Rikako
authors.initials: R
authors.lastname: Hiramatsu
authors.forename: Eiko
authors.initials: E
authors.lastname: Hasegawa
authors.forename: Tatsuya
authors.initials: T
authors.lastname: Suwabe
authors.forename: Noriko
authors.initials: N
authors.lastname: Hayami
authors.forename: Masayuki
authors.initials: M
authors.lastname: Yamanouchi
authors.forename: Fumi
authors.initials: F
authors.lastname: Takemoto
authors.forename: Shuichi
authors.initials: S
authors.lastname: Taniguchi
authors.forename: Kenmei
authors.initials: K
authors.lastname: Takaichi
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000150:complications / Q000188:drug therapy*; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage; D015331:Cohort Studies; D003131:Combined Modality Therapy; D003907:Dexamethasone / Q000008:administration & dosage; D004317:Doxorubicin / Q000008:administration & dosage; D005260:Female; D006331:Heart Diseases / Q000188:drug therapy / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D000075363:Immunoglobulin Light-chain Amyloidosis; D008107:Liver Diseases / Q000209:etiology; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D011379:Prognosis; D015996:Survival Rate; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D014750:Vincristine; D003907:Dexamethasone; D004317:Doxorubicin; D008558:Melphalan